Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia.

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01305694
• Date of registration: 28/02/2011
• Prospective Registration: No
• Primary sponsor: Guangzhou General Hospital of Guangzhou Military Command
• Public title: Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia. MSC
• Scientific title: Phase?/?Trial of Bone Marrow Derived Mesenchymal Stem Cell Transplantation From Related Donor to Patients With Relapsed/Refractory Aplastic Anemia.
• Date of first enrolment: February 2011
• Target sample size: 50
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT01305694
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

China

Contacts

• Name: Yang Xiao, MD

• Name: Yang Xiao, MD

• Name: Yang Xiao, MD
• Affiliation: Guangzhou General Hospital of Guangzhou Military Command

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients must fulfill definition of aplastic anaemia:

There must be at least two of the following:

haemoglobin < 100g/L; platelet count < 50 x 109/L; neutrophil count < 1.5 x 109/L, and a
hypocellular bone marrow;

SAA as defined by a hypocellular bone marrow of <25% cellularity and two of the following:

neutrophil count < 0.5 x 109/L platelets < 20 x 109/L reticulocytes < 20 x 109/L nSAA as
defined by a hypocellular bone marrow and cytopenia in at least two cell lines and
neutrophil count > 0.5 x 109/L, and red cell and/or platelet transfusion dependence.

- Patients belong to acquired aplastic anaemia.

- Patients with a history SAA must have had an incomplete response at least 3 months
following treatment with ATG/CsA, or they must have relapsed following an initial
response to treatment, and they do not have a HLA-matched donor for bone marrow
transplantation. Patients with a history nSAA must have red cell and/or platelet
transfusion dependence.

- Peripheral blood counts at the time of enrollment must include at least one of the
following: haemoglobin < 90 g/L or red blood cell (RBC) transfusion dependence, PMN <
1 x 109/L, or platelet count < 50 x 109/L.

- Patients must have organ function as defined below:

total bilirubin within normal institutional limits (NV: 0.0-20.5 umol/L)
AST(SGOT)/ALT(SGPT) < 2.5 × institutional upper limit of normal AST (NV: 0-35 U/L); ALT
(NV: 0-40 U/L) Creatinine within normal institutional limits (NV: 53-106 umol/L) or
Creatinine clearance > 1.25 ml/s for patients with creatinine levels above institutional
normal.

- Age minimum 16 years old with no upper age limit.

- Ability to understand and the willingness to sign a written informed consent
document.

Exclusion Criteria:

- Patients may not be receiving any other investigational agents within 4 weeks of
study entry.

- History of allergic reactions attributed to compounds of similar biologic composition
to mesenchymal stem cells.

- Current diagnosis of Fanconi's anemia, Dyskeratosis Congenita (DC) or other
hereditary forms of AA.

- Psychiatric, addictive or any other disorder that compromises ability to give a truly
informed consent.

- Age < 16 years old.

- ECOG performance status > 2.

- Malignancy within the last 5 years.

- Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection (defined as invasive fungal infection and progressive CMV viremia),
symptomatic congestive heart failure (NYH class III and IV), unstable angina
pectoris, or cardiac arrhythmia.

- Pregnant or breastfeeding women.

- HIV-positive patients.



Age minimum: 16 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Biological: bone marrow derived mesenchymal stem cells

Primary Outcome(s)

Number of participants with adverse events [Time Frame: up to 30 days]

Secondary Outcome(s)

Clonal evolution to PNH, myelodysplasia or acute leukemia [Time Frame: up to 1 year]

Hematologic response [Time Frame: up to 1 year]

Relapse [Time Frame: up to 1 year]

Survival [Time Frame: up to 1 year]

Secondary ID(s)

HM-2010-16

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Guangzhou Municipal Twelfth People's Hospital

Guangdong Prevention and Treatment Center for Occupational Diseases